Abstract
AAV-mediated gene therapy (GT) is a promising new treatment for hemophilia B patients. In contrast with the standard treatments that rely on replacing the missing clotting factor, either on-demand (OD) or by regular prophylactic infusions (Pro), GT provides endogenous factor production long-term after a single infusion. Patients who have factor inhibitors or neutralizing AAV-antibodies are mostly not eligible to receive GT and are thus often excluded from cost-effectiveness analyses. Our study examined the cost-effectiveness of GT compared to alternative treatments in two target populations, the GT-eligible cohort versus the total hemophilia B population.
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